Safety and Efficacy Study of Sivelestat in Neuromyelitis Optica Spectrum Disorder

NCT07738952 | Not Yet Recruiting Phase 1

• 1 other identifier: CTR542-01
• Study Type: interventional
• Target: 7
• Locations: 0 countries
• Sites: N/A

Brief Summary

The primary objective of this study is to evaluate the safety and tolerability of sivelestat sodium hydrate administered in combination with standard steroid pulse therapy in patients experiencing an acute NMOSD attack. Safety assessments will include adverse events, laboratory parameters, vital signs, and other clinically relevant findings. In addition, the study will explore whether the addition of sivelestat sodium hydrate to standard steroid pulse therapy improves neurological outcomes in patients with acute NMOSD. Participants will receive intravenous sivelestat sodium hydrate at a dose of 4.8 mg/kg/day administered as a continuous infusion (0.2 mg/kg/hour) for 5 consecutive days, receive steroid pulse therapy according to the study protocol, and be followed for 28 days after treatment initiation for safety and efficacy evaluations.

Conditions

Neuromyelitis Optica Spectrum Disorder Attack

Keywords

NMOSD; Sivelestat

Outcome Measures

Primary Outcomes (1)

• Incidence of adverse events

  4 weeks

Secondary Outcomes (14)

• Change from baseline in body temperature at 3 hours, Day 2, Day 3, Day 4, Day 5, and Day 6 after the start of investigational product administration.

  6 days

• Change from baseline in blood pressure at 3 hours, Day 2, Day 3, Day 4, Day 5, and Day 6 after the start of investigational product administration.

  6 days

• Change from baseline in pulse rate at 3 hours, Day 2, Day 3, Day 4, Day 5, and Day 6 after the start of investigational product administration.

  6 days

• Change from baseline in percutaneous arterial oxygen saturation at 3 hours, Day 2, Day 3, Day 4, Day 5, and Day 6 after the start of investigational product administration.

  6 days

• Change from baseline in Expanded Disability Status Scale (EDSS) score at 4 weeks (Day 28).

  4 weeks

Study Arms (1)

Sivelestat

EXPERIMENTAL

Drug: Sivelestat sodium hydrate

Interventions

Sivelestat sodium hydrate DRUG

Sivelestat sodium hydrate is administered intravenously at a dose of 4.8 mg/kg/day as a continuous infusion (0.2 mg/kg/hour) for 5 consecutive days in combination with steroid pulse therapy in patients with acute NMOSD attacks.

Sivelestat

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients diagnosed with anti-AQP4 antibody-positive NMOSD according to the international diagnostic criteria for NMOSD (Wingerchuk, Neurology 2015).
• Patients experiencing a relapse including any of the following, with the relapse occurring within 14 days of obtaining consent:
• i. Unilateral or bilateral optic neuritis ii. Myelitis
• Patients whose FS domain has worsened by at least 1 point due to relapse.
• Patients with one or more relapse lesions identified on MRI (however, if the relapse is considered to have occurred in the same location as an existing MRI lesion neurologically, identification of a new relapse lesion is not required).
• Patients aged 18 years or older at the time of obtaining consent.
• Female patients of childbearing potential who agree to use appropriate contraception from the time of obtaining consent until 180 days after the end of investigational product administration.
• Male patients who agree to use appropriate contraception until 90 days after the end of investigational product administration.
• Patients who can provide written informed consent.

You may not qualify if:

• Patients with multi-organ dysfunction involving 4 or more organs.
• Patients with severe chronic respiratory disease.
• Patients with autoimmune diseases other than NMOSD that are expected to require additional treatment during the study period.
• Patients with active systemic bacterial, viral, or fungal infections.
• Patients who have received either or both of the following prior treatments after an NMOSD relapse:
• i. Two or more courses of steroid pulse therapy ii. Plasmapheresis iii. High-dose immunoglobulin therapy
• Patients with severe hepatic dysfunction.
• Patients with alcohol dependence, drug dependence, or psychiatric disorders that would interfere with study participation.
• Patients who have received other investigational drugs within 3 months prior to obtaining consent.
• Pregnant women, women suspected of being pregnant, or breastfeeding women.
• Patients with allergies to the investigational product or concomitant medications.
• Patients with severe allergies or a history of severe allergies.
• Patients with suicidal tendencies meeting any of the following criteria:
• Other patients judged inappropriate by the principal investigator or sub-investigator.

Sponsors & Collaborators

• Kyushu University: lead

Central Study Contacts

Mitsuru Watanabe, MD, PhD

CONTACT

+81926425340; shinkein@med.kyushu-u.ac.jp

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Lecturer

Study Record Dates

• First Submitted: July 17, 2026
• First Posted: July 31, 2026
• Study Start (Estimated): August 17, 2026
• Primary Completion (Estimated): January 31, 2028
• Study Completion (Estimated): May 31, 2028
• Last Updated: July 31, 2026

Record last verified: 2026-07

Data Sharing

• IPD Sharing: Will not share